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A Study of ROC-101 Hydrochloride for Patients with Pulmonary Arterial Hypertension and Pulmonary Hypertension Associated with Interstitial Lung Disease

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study involves two types of lung conditions that affect blood vessels in the lungs. The first condition is Pulmonary Arterial Hypertension, which means high blood pressure in the arteries that carry blood from the heart to the lungs. The second condition is Pulmonary Hypertension Associated with Interstitial Lung Disease, which is high blood pressure in the lung arteries that occurs together with scarring and stiffness of the lung tissue. The study will test a medication called ROC-101, which is given as a tablet by mouth. Participants will receive ROC-101 in addition to their current standard treatments for their condition.

The purpose of the study is to evaluate how ROC-101 affects the resistance to blood flow in the lung blood vessels and to determine if the medication is safe and well tolerated when given by mouth. The study will also look at other measures of how well the heart and lungs are working. During the study, participants will continue taking their usual medications for their lung condition while also taking ROC-101 for up to 24 weeks. Some participants may be able to continue in an extension period beyond 24 weeks. The study will measure changes in lung blood vessel resistance using a procedure called right heart catheterization, which involves inserting a thin tube into the heart through a blood vessel to measure pressures and blood flow.

Throughout the study, participants will have regular visits where doctors will check their safety through physical examinations, blood tests, heart tracings, and monitoring for any side effects. The study will also measure how far participants can walk in six minutes, check levels of a substance in the blood called NT-proBNP that indicates heart stress, and assess symptom severity. Additional measurements will include various heart and lung pressures, oxygen levels in the blood, and how much blood the heart pumps with each beat. Participants will need to use effective birth control methods during the study and for a period after stopping the medication.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Screening and baseline assessments
Before starting treatment, a right heart catheterization will be performed within 2 weeks of or during the screening period. This is a procedure that measures pressures in the heart and lungs using a thin tube inserted through a blood vessel.
A ventilation-perfusion scan (or similar imaging test) will be conducted if applicable to the condition. This test shows how air and blood flow through the lungs.
A 6-minute walk test will be performed twice, once during screening and once at baseline. This test measures how far the body can walk in 6 minutes.
An electrocardiogram will be recorded at screening and on day 1 to check the heart's electrical activity.
Blood tests, including NT-proBNP (a marker that indicates heart strain), will be collected.
Lung function tests will be performed to measure breathing capacity.
Body weight and height will be measured to ensure weight is greater than 50 kilograms and body mass index is between 19 and 36.
For women of childbearing potential, two negative pregnancy tests will be required before starting the study drug.
Step 2
Starting treatment with ROC-101
Treatment with ROC-101 tablets will begin on day 1 after all baseline assessments are completed.
The medication will be taken by mouth.
Background therapy for the condition must remain stable. If already taking medications for pulmonary hypertension, these should have been at a stable dose for at least 30 days before starting ROC-101.
The tablets must be swallowed whole.
Step 3
Main treatment period
The main treatment period will last 24 weeks.
Throughout this period, regular visits will be scheduled to monitor safety and effectiveness.
Safety assessments will include monitoring for any unwanted effects, measuring vital signs such as body temperature, heart rate, blood pressure, breathing rate, and oxygen levels.
Blood tests and electrocardiograms will be performed at scheduled intervals.
The 6-minute walk test will be repeated to assess changes in walking distance.
Blood samples will be collected to measure NT-proBNP levels.
Assessment of symptoms will be performed using the World Health Organization functional class, which categorizes the severity of symptoms during physical activity.
Step 4
Week 24 assessment
At week 24, a right heart catheterization will be performed again to measure changes in lung blood vessel resistance and other heart and lung pressures.
The 6-minute walk test will be performed to compare walking distance with baseline.
Blood tests including NT-proBNP will be collected.
Symptom assessment using the World Health Organization functional class will be conducted.
All safety assessments will be completed, including vital signs, electrocardiogram, and laboratory tests.
Step 5
Extension period (optional)
After completing the main 24-week treatment period, there may be an option to continue in an extension period.
To enter the extension period, all assessments through the week 24 visit must be completed.
Women of childbearing potential must have a negative pregnancy test.
Contraceptive measures must continue for 28 days after the last dose for women of childbearing potential and 90 days after the last dose for men.
Treatment with ROC-101 tablets will continue during this period.
Regular monitoring visits will continue with safety assessments and effectiveness evaluations.

Who can join the trial?

20 criteria

You must be 18 years of age or older when signing the consent form
You must understand and voluntarily sign the informed consent form, which is a document explaining the study details, before any study procedures begin
You must have a confirmed diagnosis of either Pulmonary Arterial Hypertension (high blood pressure in the arteries of the lungs) belonging to specific types, or Pulmonary Hypertension Associated with Interstitial Lung Disease (high blood pressure in lung arteries combined with scarring of lung tissue), confirmed by a right heart catheterization, which is a procedure where a thin tube is inserted into your heart to measure pressures
You must have symptoms classified as WHO Functional Class II or III, which means you have some limitations in your physical activity due to your condition
Your right heart catheterization results must show specific pressure measurements: pulmonary vascular resistance (resistance to blood flow in lung vessels) of at least 5 Wood units for some patient groups or at least 3 Wood units for lung disease patients, pulmonary capillary wedge pressure (pressure in small lung blood vessels) of 15 mmHg or less, and mean pulmonary artery pressure (average pressure in the main lung artery) greater than 20 mmHg
If you have Pulmonary Arterial Hypertension, you must be taking at least two approved medications for your condition for at least 90 days before screening, with stable doses for at least 30 days before starting the study
If you have Interstitial Lung Disease with pulmonary hypertension, you may be taking certain inhaled or oral medications, but not other specific types of treatments
If you are a woman who can become pregnant, you must have two negative pregnancy tests before starting the study medication and agree to use highly effective birth control methods for at least 28 days before starting, during the study, and for 28 days after stopping the study medication
If you are a woman who can become pregnant, you must not breastfeed or donate eggs during the study and for at least 28 days after the last dose
If you are a male participant, you must have had a vasectomy (surgical procedure to prevent pregnancy) with confirmed absence of sperm at least 120 days before screening, and also use barrier birth control during the study and for at least 90 days after the last dose
You must be able to communicate well with the study team and understand the study procedures
You must be able to swallow tablets
Your pulmonary function tests (breathing tests) must show specific results depending on your condition type, measuring how well your lungs are working
If you have Pulmonary Arterial Hypertension, you must have a ventilation-perfusion scan (imaging test showing air and blood flow in lungs) or similar test showing normal or low probability results that are not clinically significant
Your electrocardiogram (heart rhythm test) must show acceptable findings including normal heart rhythm between 40 and 100 beats per minute and specific measurements within normal ranges
Your body weight must be greater than 50 kilograms and your body mass index (calculation using height and weight) must be between 19.00 and 36.00
You must be able to walk between 100 and 550 meters in a 6-minute walk test, performed twice with results within 15% of each other
For the extension period, you must complete the main study period through the Week 24 visit
For the extension period, if you are a woman who can become pregnant, you must have a negative pregnancy test
For the extension period, all participants must follow birth control guidance until 28 days after the last dose for women who can become pregnant and 90 days after the last dose for males

Who cannot join the trial?

4 criteria

The source data does not provide specific exclusion criteria, which are the reasons why a patient cannot participate in the study
Without detailed exclusion criteria information, it is not possible to list the specific conditions or situations that would prevent participation in this clinical trial
Exclusion criteria typically include things like certain other medical conditions, medications being taken, test results, or other factors that might make the study unsafe or affect the results
To learn about the specific reasons you may not be able to participate in this study, you would need to review the complete study documentation

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Verified
Azienda Ospedaliero Universitaria Ospedali Riuniti
Foggia, Italy
Verified
Fondazione IRCCS Policlinico San Matteo
Pavia, Italy
Responsive
Universita Degli Studi Di Roma La Sapienza
Rome, Italy

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Investigated drugs

ROC-101 is an oral medication being tested in this study. It is given by mouth to patients with pulmonary arterial hypertension and pulmonary hypertension associated with interstitial lung disease. This medication is being studied to see if it can help reduce resistance in the blood vessels of the lungs and to evaluate how safe it is when used alongside the patient's current standard treatments.

ROC-101 HYDROCHLORIDE

What is already known about the treatment

ROC-101 – ROC-101 is an oral medication currently being studied in Phase 2A clinical trials for the treatment of Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated with Interstitial Lung Disease (ILD-PH). The medication is taken by mouth and is being tested to see if it can reduce the resistance in blood vessels of the lungs (pulmonary vascular resistance), which would make it easier for the heart to pump blood through the lungs. The study is evaluating whether ROC-101 is safe and well-tolerated when used together with standard treatments, and whether it can effectively improve the condition of patients with these types of lung-related high blood pressure disorders.

Investigated diseases

Pulmonary Arterial Hypertension
Pulmonary arterial hypertension is a condition where the blood pressure in the arteries of the lungs becomes abnormally high. The small blood vessels in the lungs become narrowed, blocked, or damaged over time. This makes it harder for blood to flow through the lungs, forcing the heart to work harder to pump blood. As the disease progresses, the right side of the heart becomes weakened from the extra effort. People with this condition may experience shortness of breath, fatigue, chest pain, and dizziness. The symptoms typically worsen over time as the blood vessels continue to narrow and the heart struggles more to maintain adequate blood flow.
Pulmonary Hypertension Associated with Interstitial Lung Disease
This condition occurs when high blood pressure develops in the lung arteries as a result of scarring or damage to the lung tissue itself. Interstitial lung disease causes the tissue between the air sacs in the lungs to become thick and stiff. As the lung tissue becomes damaged, the blood vessels in the lungs are also affected, leading to increased pressure. The combination of damaged lung tissue and high blood pressure in the lung vessels makes breathing increasingly difficult. The condition progresses as both the lung scarring and the vessel pressure worsen over time. Symptoms include severe shortness of breath, persistent cough, and reduced ability to perform physical activities.